Cystic Fibrosis Notes and Notable – Robert K. Coughlin, President & CEO, Massachusetts Biotechnology Council
Drug Market Info presents Cystic Fibrosis Notes & Notable, an interview series with leading voices in the fight to cure Cystic Fibrosis.
Today’s interview is with Robert K. Coughlin, the President & CEO of Massachusetts Biotechnology Council and the father of a son with Cystic Fibrosis. Bob is a major figure in the biotech community, a devoted father and major fundraiser for Cystic Fibrosis. He was asked to share his personal experiences and provide a perspective on drug development. Here are his comments:
Can you tell us a little bit about your son’s experience with CF and your family’s experience as caregivers?
Bobby is our third child and is currently nine years old. Our older two children were healthy and thriving. We found out in utero (21 weeks into the pregnancy) that he was affected with Cystic Fibrosis. Mom was screened for the mutation for this pregnancy and not the prior two. When we found out Mom was a carrier, I was screened and also found to be a carrier of the CF mutation. We then proceeded to test our unborn child through DNA testing of the amniotic fluid. We learned we both passed off the mutation and he would be born with CF. This knowledge enabled us to treat the disease immediately. We had the opportunity to grieve prior to him joining us and we have had a positive attitude since the beginning.
What are the daily struggles and triumphs? How do you measure or evaluate his health/progress?
Bobby’s life is not easy. He takes many meds, has trouble digesting food and struggles to gain weight. He is a very outgoing little boy that doesn’t let it hold him back. He plays hockey and baseball and loves to stay active. Overall he has been relatively healthy for a CF patient. He has only been hospitalized once for a clean out and most of his therapies we can administer at home.
On a typical day, how many medications does your son use? How much time does this involve out of his day?
Bobby often says that the worst part about having CF is the amount of time he needs to spend taking nebulized meds and receiving chest physical therapy. He gets chest PT three to five days per week. These sessions take approximately an hour for the therapist to manually “clap” various areas of his chest to loosen mucous from the lungs. He also needs to take two to three nebulized meds per day and each session takes from 30 to 45 minutes. He gets really sad sometimes when other kids are outside playing and he needs to dedicate this time to staying healthy. It is difficult for a 9-year-old, active boy to understand this but he mostly stays positive.
He has to take 3-5 pancreatic enzyme pills with every meal and snack. Some days he takes up to 50 pills per day just to eat food. These enzymes don’t work well much of the time and he has very uncomfortable abdominal pains. He also takes vitamins A, D, E, C orally as well as some other supplements. Lastly, he is on a medicine that he takes twice a day to help keep the swelling of his liver under control.
What improvements are needed in these therapies that might make them easier to take?
As parents and advocates, we always encourage the formulation of both better drugs and more efficient delivery modes. Bobby would love nebulized meds to be done “really fast” so he can get out and play. Living life is important to anyone facing chronic illness. The only pancreatic enzymes available are animal-based and deteriorate over time due to temperature changes. There needs to be a synthetic solution that can be dosed in a way to reduce the pills needed every day.
What are your thoughts on the current state of CF therapies available to patients?
We have decided to continue with manual chest PT as opposed to using a mechanical vest for this therapy. We feel that the current vests are not as effective. Until recently, most of the CF medicines were antibiotics to fight infection in the lungs. This is not a sustainable treatment due to the damage caused during infection and the bacteria’s ability to build up a resistance and stop working as the patient gets older. We have desired for many years that a synthetic enzyme be invented as well as a therapy that would fix the defect as opposed to treating the symptoms of the disease.
Do you know what CF mutation your son has? If yes, when/how did you learn of this?
My son has the deltaF508 mutation from me and the 1717-1 from his Mom. We learned of this before he was born.
How will the recent approval of Kalydeco (Vertex) change the treatment of CF for those who are able to use it?
The approval of Kalydeco is going to and is currently changing the world for patients with the G551D mutation. They may no longer be required to take all of the other therapies and hopefully the physical deterioration will be stopped in its tracks! The work is not done and we strongly support and advocate for the follow-on products that will enable Kalydeco to work for all of the CF mutations.
Do you think there are enough companies pursuing drug development for CF?
For rare diseases, there is often not enough research being done. We could argue that if there is an unmet medical need, you need to do more and more research. However, I feel that the Cystic Fibrosis Foundation has rallied with the Cystic Fibrosis community for many years to raise both awareness and research dollars. We have been and always will continue to raise money and ultimately find a cure for all who suffer from this horrible disease.
About Robert Coughlin
Bob Coughlin is President and CEO of Massachusetts Biotechnology Council. He has spent his career in both the public and private sectors, most recently serving as Undersecretary of Economic Development within Massachusetts Governor Deval Patrick’s administration. He was elected as State Representative for three terms where both healthcare and economic development were his priorities.
Bob has served as the honorary chairman of the Great Strides Cystic Fibrosis Walk and has co-chaired the Children’s Hospital Boston signature event, Champions for Children’s. His son Bobby has Cystic Fibrosis and is 9 years old.
Looking for the facts about Cystic Fibrosis and the Cystic Fibrosis market? The following information comes from our Cystic Fibrosis Market Info™ Report which is now available for purchase and download on our website, Drug Market Info.
Posted on April 30, 2012, in Cystic Fibrosis, Notes and Notables, Patients' Perspective and tagged CF, cf mutation, cf patients, Cystic Fibrosis, Cystic Fibrosis Foundation, Cystic Fibrosis Market Info, Drug Market Info, Kalydeco, massachusetts biotechnology council, patients perspective, Robert Coughlin. Bookmark the permalink. 1 Comment.